400 Section - A retrospective cohort study assessing persistence to botulinum toxin A and factors associated with discontinuation

A retrospective cohort study assessing persistence to botulinum toxin and factors associated with discontinuation
Michael Wainberg,1* Jinming Zhang,2 Simon Page,3 Nivedita Rangarajan,4 Sandeep Kumar,4 Jonathan Bouchard,2 Amandeep Mann,2 Mathieu Beneteau,5 Billie Schultz1
Affiliations:
1Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, Minnesota;
2Ipsen, Cambridge, United States;
3Ipsen, London, United Kingdom;
4nference, Cambridge, MA, USA;
5Ipsen, Paris, France

*Corresponding author: Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, Minnesota, 200 First St. SW, Rochester, MN 55905, USA.
Email address: wainberg.michael@mayo.edu

Topic/Theme: Clinical sciences/health conditions
Subtopic: Regenerative Rehabilitation

Objectives: To investigate real-world botulinum toxin (BoNT) treatment persistence in patients with neurological disorders.

Design: Electronic health records (January 2010–June 2023) were used to characterize BoNT persistence in adult patients with ≥1 treatment cycle, overall and across mutually-exclusive International Classification of Diseases 9/10-defined cohorts: stroke, migraine, bladder dysfunction (BD), cervical dystonia (CD), and adult cerebral palsy (CP). Index date was first BoNT treatment; patients were followed for two years post-index. Persistence/non-persistence were defined by evaluating if all treatment intervals post-index were ≤80th percentile of all patients in the cohort (persistent) or if any were >80th percentile (non-persistent). Reasons for discontinuation were identified by AI natural language processing (NLP) analysis of physicians’ free-text notes, and further stratified by BoNT-A types (onabotulinumtoxinA, incobotulinumtoxinA, abobotulinumtoxinA and rimabotulinumtoxinB).

Results: Of 24,212 patients (mean age 52.3 years, 73.6% female) persistency was evaluable for 23,746; 11.1% were persistent. Cross-cohort persistency was 5.7% (stroke, 23/407), 7.4% (migraine, 408/5543), 10.5% (BD, 62/593), 10.3% (CD, 102/989), 14.8% (CP, 8/54). Persistent (vs non-persistent) patients were generally younger (stroke, BD) and more often female (stroke, BD, CD); neither trend was seen in the migraine cohort. Mean (95% confidence interval) BoNT treatment number was 3.35 (3.32–3.39) cycles overall; lowest in BD (1.99 [1.87–2.11]); highest in migraine (3.90 [3.83–3.98]). Cardiovascular comorbidities were highest for the stroke cohort; depression and anxiety were common across cohorts (17.6–35.4% and 28.5–57.4%, respectively). Cross-cohort antidepressant use ranged from 16.7–39.5%; higher among persistent (41.0%) vs non-persistent (28.4%) patients overall. Most common reasons for discontinuation across cohorts were patient- or physician-described lack of treatment effectiveness (17.4–40.4% and 12.3–32.6%, respectively); reasons for discontinuation were comparable between BoNTs.

Conclusions: BoNT persistence was low across disease cohorts over a 2-year follow-up period. Understanding drivers of discontinuation may inform strategies to manage BoNT persistence.

Funding: Ipsen.

Key words (maximum 6): Botulinum toxin; persistency; stroke; cervical dystonia; cerebral palsy; treatment discontinuation